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The Value of Mouse Models of Rare Diseases: A Spanish Experience
Silvia Murillo-Cuesta1,2,3, Rafael Artuch1,4, Fernando Asensio5
1Biomedical Research Networking Center on Rare Diseases (CIBERER), Institute of Health Carlos III, Madrid, Spain.
Abstract:
Animal models are invaluable for biomedical research, especially in the context of rare diseases, which have a very low prevalence and are often complex. Concretely mouse models provide key information on rare disease mechanisms and therapeutic strategies that cannot be obtained by using only alternative methods, and greatly contribute to accelerate the development of new therapeutic options for rare diseases. Despite this, the use of experimental animals remains controversial. The combination of respectful management, ethical laws and transparency regarding animal experimentation contributes to improve society's opinion about biomedical research and positively impacts on research quality, which eventually also benefits patients. Here we present examples of current advances in preclinical research in rare diseases using mouse models, together with our perspective on future directions and challenges.
Insights
Mouse models are crucial for understanding rare disease mechanisms and developing new therapies. Ethical animal research practices improve public perception and research quality, benefiting patients.
Area of Science:
- Biomedical Research
- Rare Diseases
- Animal Models
Background:
- Rare diseases are complex and have low prevalence, making them difficult to study.
- Mouse models offer unique insights into rare disease mechanisms and therapeutic strategies.
- Animal experimentation in research remains a subject of public controversy.
Purpose of the Study:
- To highlight the importance of mouse models in rare disease research.
- To discuss advances in preclinical research for rare diseases using mouse models.
- To present perspectives on future directions and challenges in the field.
Main Methods:
- Utilizing mouse models for preclinical research in rare diseases.
- Reviewing current advances and case examples.
- Analyzing ethical considerations and societal impact of animal experimentation.
Main Results:
- Mouse models provide essential data for understanding rare disease pathology.
- Preclinical research using mouse models accelerates the development of novel therapeutic options.
- Ethical management, legal frameworks, and transparency enhance public trust and research integrity.
Conclusions:
- Mouse models are indispensable tools for rare disease research, offering critical insights unattainable through alternative methods.
- Responsible and ethical animal research practices are vital for scientific progress and public acceptance.
- Continued advancements in mouse models and ethical considerations will drive future therapeutic breakthroughs for rare diseases.
